FibroGen Completes Enrollment for Second Phase 3 Trial of Pamrevlumab

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by Patricia Inácio, PhD |

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FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a potential treatment for children with Duchenne muscular dystrophy (DMD).

The LELANTOS-2 study (NCT04632940) has enrolled 73 boys with DMD, ages 6 to 12, who maintain their ability to walk.

The trial, the second Phase 3 study of pamrevlumab, is underway at 47 clinical sites across the U.S., Italy, the Netherlands, Spain and the U.K., and follows the decision by the U.S. Food and Drug Administration to grant pamrevlumab fast track designation.

“We are very pleased to complete enrollment of our second Phase 3 study of pamrevlumab in patients with DMD,” Mark Eisner, MD, chief medical officer at FibroGen, said in a press release.

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“Pediatric patients living with DMD have limited treatment options, and LELANTOS-2 will evaluate pamrevlumab as a potential new treatment. On behalf of the entire study team, we would like to extend our gratitude to the patients, caregivers, investigators and study staff for commitment to this study,” Eisner said.

In LELANTOS-2, participants will be randomly assigned to pamrevlumab or a placebo, which will be administered along with oral corticosteroids, delivered by infusion into the bloodstream every two weeks for up to one year.

The main goal is to determine changes in motor function after 52 weeks (one year) compared to at the study’s start. This will be evaluated using the North Star Ambulatory Assessment (NSAA), commonly used to monitor disease progression and the effectiveness of treatments.

Secondary goals include tests of muscle function, such as the ability to stand, climb stairs, and run or walk a distance of 10 meters (about 11 yards). The time until loss of the ability to walk will be assessed.

Top-line data from LELANTOS-2 is expected by the second half of 2023.

The first Phase 3 trial, LELANTOS-1 (NCT04371666) is evaluating pamrevlumab (also in combination with corticosteroids)  in DMD boys, ages 12 and older, who are no longer able to walk. Its main goal is to determine changes in upper limb function after 52 weeks.

According to FibroGen, top-line data from LELANTOS-1 expected in the first half of 2023.

Pamrevlumab is an antibody designed to target the connective tissue growth factor (CTGF), an inflammatory protein that causes muscle scarring. CTGF levels are abnormally high in Duchenne patients, leading to muscle weakness and organ impairment.